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Acute graft-versus-host disease is a common complication of allogeneic hematopoietic stem cell transplantation and is a major cause of morbidity and mortality. Systemic steroid therapy is the first-line treatment for acute graft-versus-host disease, although about half of patients will become refractory to treatment. We aimed to evaluate treatment options by reviewing available alternatives for patients with steroid-refractory acute graft-versus-host disease by comparing data from recently published studies. We retrospectively studied the safety and efficacy of treatment in 22 children with steroid‐dependent/refractory acute GVHD between the years 2010 and 2023. Seven (31.8%) out of 22 patients with acute graft-versus-host disease were still alive. The seven surviving patients have been followed for an average of 1141 (±403) days. 15 non-responders with grade III/IV acute graft-versus-host disease died from causes associated with acute graft-versus-host disease and/or other conditions. Among 15 patients who died, the number of patients who received all three treatments, MSC, ECP and infliximab, was 5 (33%). The most common cause of death was infection (8 cases in 15 patients, 53.3%). Other causes of death were gastrointestinal hemorrhages (n=5, 33.3%), and intracranial hemorrhages (n=2, 13.3%). When factors causing morbidity were evaluated, it was observed that three patients developed posterior reversible encephalopathy syndrome, two patients developed hepatic veno-occlusive disease, and one patient developed hypertension. The estimated probability of survival after 1 year was 31.8%, and the median survival was 655 days. We believe that it would be crucial to show the safety and efficacy of novel treatments in comprehensive, randomized clinical trials.
Aygüneş et al. (Thu,) studied this question.